Early success of LentiGlobin in trial for sickle cell disease- bluebirdbio Inc.

bluebird bio, Inc., a clinical-stage company committed to developing potentially transformative gene therapies for severe genetic and rare diseases and T cell-based immunotherapies, has announced that data from the ongoing Phase II HGB-205 study of LentiGlobin BB305 Drug Product will be presented in an oral presentation on June 13, 2015 at the 20th Congress of the European Hematology Association (EHA) in Vienna, Austria.

This subject (1204) entered the trial receiving chronic transfusions and began the process of being weaned from transfusions after day 37, receiving the last transfusion on day 88.Increasing production of HbAT87Q; the first-ever SCD patient treated with gene therapy (subject 1204) had a HbAT87Q level of 24% at 4.5 months follow up, compared to an HbAT87Q level of 9.6% at three months post-transplant Note that this subject did not engraft until after month one, so their level of HbAT87Q production at months three and 4.5 are actually months two and 3.5, after engraftmentAt 4.5 months follow up, total anti-sickling hemoglobin (HbAT87Q + HbF) was 31.6%.

Subject 1204 has not had any hospitalizations for SCD-related complications post-transplatn. No subject has experienced a drug product-related adverse event, and integration site analyses demonstrate highly polyclonal reconstitution without clonal dominance.Based on historical clinical observations in patients with SCD, bluebird bio believes that individuals who achieve more than 30 percent of anti-sickling hemoglobin (HbAT87Q + HbF) have the potential to reduce or eliminate the serious and life-threatening events associated with SCD.

Comment : LentiGlobin is also in trials for beta-thalassemia.